Gene Therapy

Orphan (or rare) diseases

While the term “orphan disease” implies it is rare in occurrence, the impact on the patients and families are long-term and in some cases, fatal. Much of bluebird’s core work focuses on three rare disease states: Adrenoleukodystrophy, β-thalassemia and sickle cell disease.

Sickle Cell Disease

Product Candidate

At bluebird, we’re making real and exciting progress, especially with two separate product candidates – Lenti-D, a potential treatment for CALD, and LentiGlobin, a potential treatment therapy for TDT and SCD.